NGGT002

NGGT002 is an investigational gene therapy designed for the treatment of phenylketonuria (PKU), a rare inherited metabolic disorder caused by mutations in the PAH gene leading to deficient activity of phenylalanine hydroxylase. The therapy consists of a recombinant adeno-associated virus serotype 8 (rAAV8) vector that delivers a functional copy of the human PAH gene to liver cells, aiming to restore normal metabolism of phenylalanine and reduce its toxic accumulation in patients. Clinical studies have shown that high-dose administration can normalize plasma phenylalanine levels in most treated patients, with effects sustained for several weeks post-treatment. The drug is currently being evaluated in Phase I/II clinical trials in both the United States and China[1][2][4][5][6][9][10].